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Saturday, December 1, 2018

bluebird Updates β-Thalassemia, Sickle Cell Studies at ASH


All patients with transfusion-dependent β-thalassemia and a non-β00 genotype who achieved transfusion independence continue to maintain it for up to 3.5 years in Phase 1/2 Northstar (HGB-204) study
HbAT87Q levels were stable and vaso-occlusive events were reduced in most Group A and B patients with sickle cell disease in Phase 1/2 HGB-206 study following treatment with LentiGlobin
bluebird bio, Inc. (Nasdaq: BLUE) announced new long-term data from the completed Phase 1/2 Northstar (HGB-204) study of investigational LentiGlobin™ gene therapy in patients with transfusion-dependent β-thalassemia (TDT) and from the ongoing Phase 1/2 HGB-206 study of LentiGlobin in patients with sickle cell disease (SCD) today at the 60th Annual Meeting of the American Society of Hematology (ASH).
“The breadth of our LentiGlobin data at ASH across multiple clinical trials reflects the commitment of patients, families and healthcare providers to investigate the transformative therapeutic potential of gene therapy for the beta-hemoglobinopathies,” said David Davidson M.D., chief medical officer, bluebird bio. “LentiGlobin gene therapy is designed to address the underlying genetic cause of beta-thalassemia and sickle cell disease. The longer-term data emerging from our clinical trials show that most treated patients are producing sufficient amounts of engineered HbAT87Q to achieve and maintain a therapeutic benefit.”

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